Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) (EXIST-2)

This study has been completed.

Sponsor:

Novartis Pharmaceuticals

ClinicalTrials.gov Identifier:

NCT00790400

First Posted: November 13, 2008

Last Update Posted: February 17, 2017

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government.
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Key information relevant to the recruitment process for the
overall study, such as dates of the recruitment period and locations

A multicenter trial conducted at 24 sites in 11 countries. As the primary analysis of the core phase of the study favored everolimus over placebo, an open-label extension phase started: patients randomized in placebo were offered to switch on everolimus and those still receiving everolimus at the end of the core phase could continue the treatment.

Pre-Assignment Details

Significant events and approaches for the overall study
following participant enrollment, but prior to group assignment

The trial had a 2:1 randomization in favor of the everolimus arm. 118 patients were randomized to the core phase of the study. 112 patients received everolimus during core and/or extension phase.

Reporting Groups

Description

Everolimus

Study drug was given by continuous oral daily dosing of two 5 mg tablets.

Placebo

Placebo was given by continuous oral daily dosing of two 5 mg tablets.

Participant Flow for 2 periods

Period 1: Double-blind Period (Core Phase)

Everolimus

Placebo

STARTED

79

39

COMPLETED

72 [1]

26 [2]

NOT COMPLETED

7

13

Protocol Violation

1

0

Progressive disease

0

9

Adverse Event

2

4

Abnormal lab value (s)

1

0

Withdrawal by Subject

1

0

Administrative problems

1

0

Death

1

0

[1]

Completed = Completed the Core phase & moved to Extension phase

[2]

Completed = 1st received Placebo in the Core phase, switched to Everolimus in Extension phase

Period 2: Everolimus Period (Core or Extension)

Everolimus

Placebo

STARTED

112 [1]

0 [2]

COMPLETED

83 [3]

0

NOT COMPLETED

29

0

Adverse Event

9

0

Abnormal lab value (s)

1

0

Withdrawal by Subject

7

0

Lost to Follow-up

1

0

Administrative problems

2

0

Death

1

0

Disease progression

5

0

Protocol Violation

1

0

New treatment

2

0

[1]

112 pts had Everolimus during core and/or extension (6 from placebo did not switch to Eve. in ext.)

[2]

Placebo randomized patients who switched to Everolimus are reported in "Everolimus" arm.

Explanation of how the number of participants for analysis was determined.
Includes whether analysis was per protocol, intention to treat, or another method.
Also provides relevant details such as imputation technique, as appropriate.

No text entered.

Reporting Groups

Description

Everolimus

Study drug was given by continuous oral daily dosing of two 5 mg tablets.

Placebo

Placebo was given by continuous oral daily dosing of two 5 mg tablets.

Angiomyolipoma Response Rate as Per Central Radiology Review [ Time Frame: From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to 5.7 years ]

Time to Angiomyolipoma Progression as Per Central Radiology Review [ Time Frame: From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to about 5.7 years ]

Skin Lesion Response Rate as Per Investigator (Only Patients With at Least One Skin Lesion at Baseline) [ Time Frame: From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to 5.7 years ]

Time to Angiomyolipoma Response - Only Everolimus Patients With Angiomyolipoma Response [ Time Frame: From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to 5.7 years ]

Duration of Angiomyolipoma Response - Only Everolimus Patients With Angiomyolipoma Response [ Time Frame: From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to about 5.7 years ]

Duration of Skin Lesion Response - Only Everolimus Patients With Best Overall Skin Lesion Response of Complete Clinical Response (CCR) or Partial Response (PR) [ Time Frame: From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to about 5.7 years ]

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts
the PI's rights to discuss or publish trial results after the trial is completed.

The agreement is:

The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release
and can embargo communications regarding trial results for a period that is less than or equal to 60 days.
The sponsor cannot require changes to the communication and cannot extend the embargo.

The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release
and can embargo communications regarding trial results for a period that is more than 60 days but less than
or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.

Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.

Restriction Description:
Principal Investigators are NOT employed by the organization sponsoring the study. Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. The terms and conditions of Novartis' agreements with its investigators may vary. Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.